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Trial record 1 of 1 for:    NCT04360265
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A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04360265
Recruitment Status : Enrolling by invitation
First Posted : April 24, 2020
Last Update Posted : February 7, 2024
Sponsor:
Collaborator:
Abeona Therapeutics, Inc
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Brief Summary:
The main objective of this study is to evaluate the long-term safety and tolerability of ABO-102 in participants with MPS IIIA.

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis III-A Other: No Investigational Product Drug: Adjuvant Immunosuppression (IS) Therapy Phase 3

Detailed Description:

This is a multicenter, long-term follow-up study of patients with MPS IIIA who have completed a prior clinical trial involving the administration of ABO-102 (NCT02716246 and NCT04088734). No investigational product will be provided. At approved sites immunosuppression (IS) therapy may be administered to selected participants. The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS therapy. Not all participants may receive IS therapy.

This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 33 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)
Actual Study Start Date : September 28, 2020
Estimated Primary Completion Date : June 2027
Estimated Study Completion Date : June 2027


Arm Intervention/treatment
Cohort 1
Participants who have completed a prior clinical trial involving the administration of ABO-102 and are able to comply with onsite scheduled visits and assessments. Select participants may receive adjuvant IS therapy.
Other: No Investigational Product
No investigational product will be administered in this follow-up trial.

Drug: Adjuvant Immunosuppression (IS) Therapy
The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS therapy.

Cohort 2
Participants who have completed a prior clinical trial involving the administration of ABO-102 and who cannot participate in Cohort 1. Participants will partake in a reduced number of assessments, performed either onsite or at home via a combination of telehealth and home healthcare visits.
Other: No Investigational Product
No investigational product will be administered in this follow-up trial.




Primary Outcome Measures :
  1. Adverse Events [ Time Frame: Up to Year 5 ]
    Long-term Product Safety as Defined by the Incidence, Type, and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).


Secondary Outcome Measures :
  1. Change From Baseline (in Prior Trial) in Cerebrospinal Fluid Heparan Sulfate (CSF HS) [ Time Frame: Up to Month 12 ]
  2. Change From Baseline (in Prior Trial) in the Cognitive Subtest of the Bayley Scales of Infant and Toddler Development -Third Edition (BSID-III) or the Kaufman Assessment Battery for Children - Second Edition (KABC-II), Based on Developmental Age [ Time Frame: Baseline, Up to Year 5 ]
  3. Change From Baseline (in Prior Trial) in the Language Subtest of the Bayley Scales of Infant and Toddler Development -Third Edition (BSID-III) or the Kaufman Assessment Battery for Children - Second Edition (KABC-II), Based on Developmental Age [ Time Frame: Baseline, Up to Year 5 ]
  4. Change From Baseline (in Prior Trial), as Assessed by the Parent Report Using the Vineland Adaptive Behavior Scale II - Survey Interview Form (VABS-II) [ Time Frame: Baseline, Up to Year 5 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Participants that have completed a prior clinical trial involving the administration of ABO-102.
  • Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule.

Exclusion Criteria:

  • Planned or current participation in another clinical trial that may confound the safety and efficacy evaluation of ABO-102 during this study.
  • Any other situation or medical condition that precludes the participant from undergoing procedures required in this study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04360265


Locations
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United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Australia, South Australia
Women's and Children's Hospital
North Adelaide, South Australia, Australia
Spain
Hospital Clínico Universitario de Santiago
Santiago De Compostela, Spain
Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc
Investigators
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Study Director: Medical Director Ultragenyx Pharmaceutical Inc
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Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT04360265    
Other Study ID Numbers: LTFU-ABO-102
2019-002979-34 ( EudraCT Number )
UX111-CL302 ( Other Identifier: Ultragenyx Pharmaceutical Inc )
First Posted: April 24, 2020    Key Record Dates
Last Update Posted: February 7, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ultragenyx Pharmaceutical Inc:
MPS IIIA
Mucopolysaccharidosis IIIA
Sanfilippo A
Additional relevant MeSH terms:
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Mucopolysaccharidoses
Mucopolysaccharidosis III
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases